Oral Pyrophosphate Absorption in PXE Disease
Primary Purpose
Pseudoxanthoma Elasticum
Status
Withdrawn
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Disodium Pyrophosphate
Sponsored by
About this trial
This is an interventional treatment trial for Pseudoxanthoma Elasticum
Eligibility Criteria
Inclusion Criteria:
- Adults (>18 yrs)
- clinically and genetically proven PXE
- Body mass index (BMI) 18,5-34,9 kg/m2
Exclusion Criteria:
- Special groups according to researchers' decision.
- Pregnancy
- No effective contraception in females in child-bearing age.
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Open label
Arm Description
Disodiumpyrophosphate, capsuled powder, First day: 30 mg/kg fasting at 08.00 and with standard mixed meal at 12.00 Second day: 50 mg/kg fasting at 08.00 and with standard mixed meal at 12.00
Outcomes
Primary Outcome Measures
Concentration area under the curve 0-t of pyrophosphate
Maximal concentration of pyrophosphate
Cmax
Timepoint of maximal pyrophosphate concentration
Tmax
Secondary Outcome Measures
Concentration area under the curve 0-t of phosphate
AUC0-t
Maximal concentration of phosphate
Cmax
Timepoint of maximal phosphate concentration
Tmax
Full Information
NCT ID
NCT04441671
First Posted
January 20, 2020
Last Updated
April 21, 2021
Sponsor
Tampere University Hospital
Collaborators
UMC Utrecht, Hungarian Academy of Sciences
1. Study Identification
Unique Protocol Identification Number
NCT04441671
Brief Title
Oral Pyrophosphate Absorption in PXE Disease
Official Title
Oral Disodiumpyrophosphate (Na2H2PPi) Absorption in Pseudoxanthoma Elasticum (PXE)
Study Type
Interventional
2. Study Status
Record Verification Date
April 2021
Overall Recruitment Status
Withdrawn
Why Stopped
Withdrawn
Study Start Date
December 8, 2020 (Actual)
Primary Completion Date
April 1, 2021 (Actual)
Study Completion Date
April 1, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Tampere University Hospital
Collaborators
UMC Utrecht, Hungarian Academy of Sciences
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
Pyrophosphate is an endogenous, non-toxic metabolite inhibiting soft tissue calcification. The aim of our study is to find optimal dosing and safety of oral disodium-PPi (Na2H2PPi). Absorption curves (pharmacokinetics), AUC0-t, Cmax and Tmax for PPi and phosphate will be provided for healthy controls and PXE-patients both fasting and with standard meal intake.
Detailed Description
Phase II oral capsulized disodium-PPi (Na2H2PPi) powder absorption study in subjects with PXE (n=8-12) will be done in the ward of Internal Medicine of Tampere University Hospital and University Medical Center Utrecht.
After a 10-hour fast at 8.00 a.m. 30 mg/kg (first day) and 50 mg/kg (second day) capsulized dose of PPi will be given with 2 dl water. At 12.00 a.m. another capsulized 30 mg/kg (first day) or 50 mg/kg (second day) single dose of PPi with a standard mixed meal (lunch) will be given with 2 dl water at the time when a subject starts eating.
Plasma and urine sampling include plasma and spot urine electrolytes, creatinine, and pyrophosphate. Plasma sampling will be done at 0, 15, 30, 60, 120 and 240 min after ingestion of PPi. Urine spot sample will be taken at 0 and 240 min after ingestion of PPi.
Physical activity is restricted. Side-effects will be recorded.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pseudoxanthoma Elasticum
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Open label
Arm Type
Experimental
Arm Description
Disodiumpyrophosphate, capsuled powder, First day: 30 mg/kg fasting at 08.00 and with standard mixed meal at 12.00 Second day: 50 mg/kg fasting at 08.00 and with standard mixed meal at 12.00
Intervention Type
Drug
Intervention Name(s)
Disodium Pyrophosphate
Other Intervention Name(s)
Na2H2PPi
Intervention Description
Absorption trial
Primary Outcome Measure Information:
Title
Concentration area under the curve 0-t of pyrophosphate
Time Frame
two days
Title
Maximal concentration of pyrophosphate
Description
Cmax
Time Frame
two days
Title
Timepoint of maximal pyrophosphate concentration
Description
Tmax
Time Frame
two days
Secondary Outcome Measure Information:
Title
Concentration area under the curve 0-t of phosphate
Description
AUC0-t
Time Frame
two days
Title
Maximal concentration of phosphate
Description
Cmax
Time Frame
two days
Title
Timepoint of maximal phosphate concentration
Description
Tmax
Time Frame
two days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Adults (>18 yrs)
clinically and genetically proven PXE
Body mass index (BMI) 18,5-34,9 kg/m2
Exclusion Criteria:
Special groups according to researchers' decision.
Pregnancy
No effective contraception in females in child-bearing age.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pasi I Nevalainen, MD, PhD
Organizational Affiliation
Tampere University Hospital
Official's Role
Principal Investigator
12. IPD Sharing Statement
Plan to Share IPD
No
IPD Sharing Plan Description
Only anonymized data can be shared
Learn more about this trial
Oral Pyrophosphate Absorption in PXE Disease
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